Allogeneic Valve Transplantation

Valves will be taken from hearts donated by organ donors, and implanted into patients who need a new heart valve.

Study Overview

• Status: Recruiting
• Conditions: Valve Disease, Heart; Valve Heart Disease; Valve Disease, Aortic
• Intervention / Treatment: Procedure: Valve transplant

Detailed Description

Heart valves in children and young adults may need replacement or repair. In many children the options for heart valve replacement do not grow as the child grows. A transplanted valve may have the option to grow with time and may reduce the need for multiple operations over a lifetime. This trial will study the outcomes of heartfelt transplant in children and young adults undergoing this procedure. The study will look at outcomes of valve transplant and any potential side effects.

• Study Type: Interventional
• Enrollment (Estimated): 4
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Sitaram Emani, MD; Phone Number: 6173557899; Email: sitaram.emani@cardio.chboston.org

Study Locations

• United States
  • Massachusetts
    • Boston, Massachusetts, United States, 02115
      • Recruiting
      • Boston Children's Hospital
      • Contact: Sitaram Emani, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child; Adult
• Accepts Healthy Volunteers: No

Description

The proposed indications include:

• Patients requiring aortic or pulmonary valve replacement.

• Pediatric patients 30 days or older and < 18 years, and adult patients 18-50 yrs.

  o Particularly patients with significant growth potential

• Insufficient options are available for valve replacement

  o Patients at risk of immunogenic bio-prosthetic valve failure

• Discussion with patient /family - option for durable valve rather than traditional prosthesis
• ABO compatible
• Patient and family able to travel to BCH within 48 hour time frame or within our organ procurement organization
• Patients that are able to maintain follow-up at BCH during the duration of the study
• Patients that are able to provide medical record authorization for 5 year follow-up

The proposed contraindications include:

• Age < 30 days
• Irreversible multisystem organ failure; or additive effects of the multiple systems affected making transplant survival unlikely
• Progressive systemic disease with early mortality (genetic/metabolic, idiopathic, syndromic)
• Morbid obesity (BMI>35)
• Diabetes mellitus with evidence of end-organ damage
• Severe chromosomal, neurologic or syndromic abnormalities
• Immunocompromised condition (DiGeorge, SCID etc.)
• Active infection
• History of endocarditis
• HIV or chronic hepatitis B or C infection
• Malignancy within 5 years prior to transplant
• Severe renal or liver failure
• Inadequate social support for post-transplant management
• Recent history of illicit drug, tobacco or alcohol abuse despite trials/assistance to stop behavior
• Evidence of large stroke with high risk for hemorrhagic conversion
• Heart transplant recipient
• Patients that are unable to follow-up during the initial 6 month window
• Patients that are unable to provide medical record authorization for 5 years

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Valve transplantation group; This arm undergoes Allogeneic Valve transplantation	Procedure: Valve transplant; Heart valves from an organ donor will be transplanted into recipient

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Valve Regurgitation	Heart valve regurgitation by echocardiogram. The degree of regurgitation is read and quantified by an echocardiologist.	One year
Valve Annulus Growth	Measurement of valve annulus via echocardiogram, CT scan and MRI. This will be quantified by whether or not the annulus continues to increase in size as the subject grows.	One Year
Leaflet Growth	Measurement of leaflet height via echocardiogram, CT scan and MRI and reviewed by an echocardiologist. This will be analyzed by whether or not the leaflets continue to grow in size, with the subject.	One Year

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Ventricular Function	Ventricular function will be visualized using echocardiogram, measured as ejection fraction as well as shortening fraction and will be determined by an echocardiologist. The factors involved will be the symmetrical squeeze of the ventricles, the ejection fraction, ventricular end systolic and diastolic volume with calculation of stroke volume to come to a surrogate of cardiac output.	One year
Survival	The number of patients that survive the initial procedure and long term outcomes.	5 years

Collaborators and Investigators

• Sponsor: Boston Children's Hospital
• Investigators: Principal Investigator: Sitaram Emani, MD, Boston Children's Hospital

Study record dates

Study Major Dates

• Study Start (Estimated): May 1, 2024
• Primary Completion (Estimated): June 1, 2027
• Study Completion (Estimated): June 1, 2027

Study Registration Dates

• First Submitted: June 21, 2023
• First Submitted That Met QC Criteria: April 25, 2024
• First Posted (Actual): April 29, 2024

Study Record Updates

• Last Update Posted (Actual): April 29, 2024
• Last Update Submitted That Met QC Criteria: April 25, 2024
• Last Verified: April 1, 2024

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Cardiovascular Diseases; Vascular Diseases; Heart Diseases; Heart Valve Diseases; Aortic Valve Disease; Aortic Diseases
• Other Study ID Numbers: RB-P00044611

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No